10-QPeriod: Q3 FY2020

ALNYLAM PHARMACEUTICALS, INC. Quarterly Report for Q3 Ended Sep 30, 2020

Filed November 5, 2020For Securities:ALNY

Summary

This 10-Q filing for Alnylam Pharmaceuticals, Inc. as of November 5, 2020, highlights significant progress in its RNA interference (RNAi) therapeutics pipeline and commercialization efforts. The company reported strong revenue growth, driven by its marketed products ONPATTRO and GIVLAARI, which saw a substantial increase in net product revenues. Alnylam is executing on its "Alnylam 2020" strategy, aiming to become a multi-product, global biopharmaceutical company. The filing details advancements in key late-stage investigational programs, including lumasiran for primary hyperoxaluria type 1 (PH1), which has received positive opinions from regulatory bodies, and vutrisiran for ATTR amyloidosis. Financially, the company has secured significant funding through a strategic collaboration with Blackstone, providing capital to advance its pipeline. While Alnylam continues to incur operating losses, consistent with its development stage, the company expresses confidence in achieving a self-sustainable financial profile. The report also addresses the ongoing impact of the COVID-19 pandemic on operations and clinical trials, emphasizing a focus on maintaining business continuity and patient access to medicines.

Financial Statements
Beta

Key Highlights

  • 1Total revenues increased by 80% to $125.9 million for the three months ended September 30, 2020, compared to $70.1 million in the prior year, driven by a 115% increase in net product revenues.
  • 2Net product revenues for ONPATTRO and GIVLAARI reached $99.2 million in Q3 2020, a significant increase from $46.1 million in Q3 2019, reflecting strong global sales expansion.
  • 3Lumasiran received a positive CHMP opinion from the EMA recommending marketing authorization for PH1, with an EC decision expected in Q4 2020. The FDA has set a Prescription Drug User Fee Act action date of December 3, 2020.
  • 4The company secured a strategic financing collaboration with Blackstone, providing up to $2.0 billion in financing, including a $1.0 billion royalty and milestone payment arrangement for inclisiran and a $750.0 million term loan.
  • 5Research and development expenses remained relatively flat year-over-year for the three-month period but increased by 7% for the nine-month period, reflecting ongoing investment in pipeline advancement.
  • 6Selling, general and administrative expenses increased by 39% and 31% for the three- and nine-month periods respectively, driven by increased headcount and investments in commercialization and potential future launches.
  • 7Alnylam continues to navigate the COVID-19 pandemic, implementing safety protocols and adapting business practices while monitoring impacts on operations and clinical trials.

Frequently Asked Questions

Alnylam reported strong growth in net product revenues for its marketed products. For the three months ended September 30, 2020, net product revenues increased by 115% to $99.2 million, compared to $46.1 million in the same period of 2019. ONPATTRO generated $82.5 million and GIVLAARI generated $16.7 million in global net product revenues for Q3 2020.

The company highlighted significant progress in its late-stage pipeline. Lumasiran for primary hyperoxaluria type 1 (PH1) received a positive opinion from the EMA's CHMP and is awaiting a decision from the European Commission. Vutrisiran is advancing in Phase 3 studies for ATTR amyloidosis, with topline results from HELIOS-A expected in early 2021. Other advancements include progress in the APOLLO-B study for patisiran in ATTR amyloidosis with cardiomyopathy and ongoing partner-led development of inclisiran.

Alnylam has secured substantial funding through a strategic financing collaboration with Blackstone, which includes up to $2.0 billion in financing. This provides capital for pipeline development, including specific funding for vutrisiran and ALN-AGT. The company also continues to generate revenue from product sales and collaborations, and believes these resources will enable it to achieve a self-sustainable financial profile without the need for future equity financing.

The company has implemented safety measures such as remote work policies and restricted travel. While it has initiated a voluntary program for employees to return to physical locations with safety protocols, the full extent of the pandemic's impact on business operations, clinical trials, and the supply chain remains uncertain. Delays in clinical trial enrollment and regulatory review timelines are potential impacts highlighted in the filing.